Experimental combo aims to boost chemo for tough blood cancers
NCT ID NCT05342584
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial is testing whether adding the targeted drug venetoclax to standard chemotherapy can safely treat adults newly diagnosed with acute myeloid leukemia or advanced myelodysplastic syndrome. About 99 participants will receive different doses of venetoclax alongside chemo drugs daunorubicin and cytarabine. The study's main focus is finding the safest dose and monitoring side effects.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Venetoclax (a targeted drug) combined with standard chemotherapy drugs daunorubicin and cytarabine
- What this could lead to
- If successful, this could lead to a more effective treatment option for adults with newly diagnosed acute myeloid leukemia or advanced myelodysplastic syndrome.
- What could go wrong
- This is an early Phase 1 trial, so the main goal is safety, not yet proof of effectiveness. The combination may cause serious side effects, and it is too soon to know if it will improve outcomes.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 99 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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May 2022
- Expected to finish
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Dec 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 75 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * New diagnosis of AML by WHO criteria. Patients with higher risk MDS (R-IPSS\>3.5) and 10% blasts or more, or proliferative (WBC ≥ 13 x 10⁹/L) CMML-2 are also eligible at the discretion of the PI. Patients having received any prior hypomethylating agent with or without BCL2 inhibitor therapy for MDS/AML are also eligible at the discretion of the PI * Patients ≥ 18 to ≤ 75 years. * Eastern Cooperative Oncology Group (ECOG) Performance Status of ≤ 2 * Adequate renal function including creatinine clearance \> 30 mL/min based on the Cockcroft Gault equation. * Adequate hepatic function including total bilirubin \< 1.5x ULN unless increase is due to Gilbert's disease or leukemic involvement, and AST and/or ALT \< 3x ULN unless considered due to leukemic involvement * Ability to understand and provide signed informed consent * Male subjects must agree to refrain from unprotected sex and sperm donation from initial study drug administration until 90 days after the last dose of study drug. Exclusion Criteria: * Patients with t(15;17) karyotypic abnormality or acute promyelocytic leukemia (FAB class M3-AML) * Subject has known active CNS involvement with AML * Patients with New York Heart Association (NYHA) Class III or IV congestive heart failure or LVEF \<45% by echocardiogram or multi-gated acquisition (MUGA) scan * Patients with a history of myocardial infarction within the last 6 months or unstable / uncontrolled angina pectoris or history of severe and/or uncontrolled ventricular arrhythmias * Patients with uncontrolled infection with human immunodeficiency virus (HIV) or active Hepatitis B or C * Patients with known dysphagia, short-gut syndrome, or other conditions that would affect the ingestion or gastrointestinal absorption of drugs administered orally. * Subject has any other significant medical or psychiatric history that in the opinion of the investigator would adversely affect participation in this study. * Subject has a white blood cell count \> 25 x 10⁹/L. (Note: Hydroxyurea and/or cytarabine (up to 2 g/m\^2 is permitted to meet this criterion.) * Nursing women, women of childbearing potential (WOCBP) with positive urine pregnancy test, or women of childbearing potential who are not willing to maintain adequate contraception. Appropriate method(s) of contraception include oral or injectable hormonal birth control, IUD, and double barrier methods (for example a condom in combination with a spermicide).
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Montefiore Einstein Cancer Center and Children's Hospital at Montefiore (CHAM)
RECRUITINGThe Bronx, New York, 10467, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can adding venetoclax make donor stem cell transplants safer for High-Risk blood cancers?
- Can an HDAC inhibitor wipe out residual leukemia cells?
- Can an experimental pill block a cancer-driving enzyme in hard-to-treat leukemia?
- Two-Drug combo targets leukemia that outsmarted its first treatment
- Tweaking donor cells may shield older transplant patients from a dangerous complication
- Can a drug and donor cells stop leukemia from returning after transplant?